Safety and Efficacy Study of LX103 Treatment of X-Linked Retinoschisis (XLRS)

X-linked Retinoschisis Clinical Trial

Official title:

An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX103 in Patients With X-Linked Retinoschisis (XLRS)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05814952
• Other study ID #: SHGH-LX103
• Status: Recruiting
• Phase: N/A
• Start date: December 13, 2022
• Est. completion date: August 2028

Study information

• Verified date: April 2023
• Source: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The goal of this study is to evaluate the safety and efficacy of LX103 treatment of X-linked retinoschisis. This study will enroll subjects aged ≥ 6 years old to receive a single unilateral intravitreal (IVT) injection of LX103 to evaluate its safety and efficacy.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: August 2028
• Est. primary completion date: August 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

1. Willing to sign the informed consent, and willing to attend follow-up visits.

2. Male individual at least 6 years of age with a diagnosis of XLRS and documented mutations in the RS1 gene.

3. The study eye must have a best-corrected E-ETDRS visual acuity letter score of less than or equal to 63.

Exclusion Criteria:

1. Any eye with disease that would interfere with the fundus examinations.

2. The study eye is receiving topical carbonic anhydrase inhibitor, or has received topical carbonic anhydrase inhibitors in the past three months.

3. The study eye has undergone intraocular surgery within six months prior to enrollment.

4. Participant has uncontrolled hypertension or diabetes.

Related Conditions & MeSH terms

• Retinoschisis
• X-linked Retinoschisis

Intervention

Genetic:
• LX103 Injection
Qualified subjects will receive a single unilateral intravitreal injection of LX103 at Day 0 in the trial.

Locations

Country	Name	City	State
China	Shanghai General Hospital, Shanghai Jiao Tong University	Shanghai

Sponsors (2)

Lead Sponsor	Collaborator
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine	Innostellar Biotherapeutics

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse events (AEs)	Incidence of adverse events (AEs) within 24 weeks of LX103 intravitreal injection at different doses	24 weeks
Primary	Incidence of serious adverse events (SAEs)	Incidence of serious adverse events (SAEs) within 24 weeks of LX103 intravitreal injection at different doses	24 weeks
Secondary	Mean change from baseline in BCVA (LogMAR) in the study eye	ETDRS visual acuity charts will be used to assess BCVA	24 weeks, 52 weeks
Secondary	Mean change from baseline in central subfield thickness (CST) in the study eye	Mean change of CST measured using Optical Coherence Tomography (OCT)	24 weeks, 52 weeks
Secondary	Change from baseline in Electroretinogram (ERG) parameters in the study eye	Change in dark-adapted 3.0 B-wave amplitude (µV)	24 weeks, 52 weeks